Hereditary blindness therapy within sight

A FORM of hereditary blindness that can rob young men of their sight has been prevented in rats using a pioneering gene therapy.

The condition, called Leber hereditary optic neuropathy, causes blindness in young adults by destroying the optic nerve. Marisol Corral-Debrinski and her colleagues at the Pierre and Marie Curie University in Paris, France, installed functional copies of the faulty ND4 gene that causes the condition into the optic neurons of young rats, using electric pulses to temporarily open the cells' surface pores.

Treated rats retained most of their optic nerve cells, whereas untreated rats lost 40 per cent within two months (American Journal of Human Genetics, DOI: 10.1016/j.ajhg.2008.08.013).

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